S100 calcium-binding protein B (S100B), chitotriosidase 1 (CHIT1), neuron-specific enolase (NSE), and amyloid-β were also investigated as potential pharmacodynamic biomarkers in SMA; however, due to the limited number of studies involving these biomarkers, it is difficult to draw a conclusion regarding the utility of these biomarkers for monitoring the response to nusinersen therapy. The gene discussed is ENO2; the disease is proximal spinal muscular atrophy.